Company Product Description Indication Status
Phase I
Rhizen Pharmaceuticals SA, of La Chaux-de-Fonds, Switzerland Tenalisib (RP-6530) PI3K delta and gamma dual inhibitor Relapsed or refractory T-cell lymphoma Results from the phase I/Ib study were published in Cancers; it was well-tolerated with favorable safety profile, and the overall response rate was 45.7% (with a 9% complete response and 37% partial response) and median duration of response was comparable to approved therapies for T-cell lymphomas
Uniqure NV, of Amsterdam AMT-130 Gene therapy Huntington disease The independent data safety monitoring board overseeing the phase I/II trial met and reviewed 90-day safety data from the first 2 patients enrolled in the trial; no significant safety concerns were noted to prevent further dosing, and the next 2 patients are now cleared for enrollment in the study
Phase II
Apeiron Biologics AG, of Vienna APN-01 Angiotensin converting enzyme 2 stimulator COVID-19 infection Data from the first patient, published in The Lancet Respiratory Medicine, show the expected observations of an adaptive immune response, a rapid loss of virus load and reduction in inflammatory mediators, and the development of high titers of neutralizing antibodies against SARS-CoV-2 leading to a significant clinical improvement of the patient treated
Beyondspring Inc., of New York Plinabulin Guanine nucleotide exchange factor stimulator; tubulin receptor antagonist Chemotherapy-induced neutropenia  In the Protective-1 and -2 studies, grade 4 neutropenia and clinical outcomes with severe neutropenia (hospitalizations, infections and sepsis) were comparable between plinabulin and pegfilgrastim; plinabulin demonstrated advantages over pegfilgrastim for all-grade thrombocytopenia and bone pain
Hansa Biopharma AB, of Lund, Sweden Idefirix (imlifidase) Cleaves IgG-antibodies Anti-GBM antibody disease (Goodpasture's disease) Results indicate that imlifidase treatment may lead to increased renal survival in patients with anti-GBM antibody disease due to rapid clearance of IgG antibodies; 6 hours after imflidase no patient had anti-GBM antibody levels above the normal range; at 6 months, 10 patients were dialysis independent (median eGFR 27 ml/min), 4 patients were dialysis dependent, while 1 patient had died (unrelated to treatment); the safety profile was concluded as being favorable
Phase III
Bristol Myers Squibb Co., of New York Opdivo (nivolumab) PD-1 inhibitor  High-risk, muscle-invasive urothelial carcinoma In an interim analysis, CheckMate-274 trial met its primary endpoints of improving disease-free survival vs. placebo in all randomized patients and in patients whose tumor cells express PD-L1 ≥1%
Genfit SA, of Lille, France Elafibranor Dual PPAR alpha/PPAR delta agonist Primary biliary cholangitis Announced the first patient first visit for its phase III study
Marinus Pharmaceuticals Inc., of Radnor, Pa. Ganaxolone Positive allosteric modulator of GABAA receptors Refractory status epilepticus Announced it has satisfied the FDA’s protocol-specific questions for the phase III trial, and the company intends to begin enrollment for registrational phase III trial 
Novavax Inc., of Gaithersburg, Md. NVX-CoV2373 Nanoparticle vaccine with the spike protein of SARS-CoV-2 and Matrix-M adjuvant COVID-19 prophylaxis Initiated trial in the U.K. to evaluate the efficacy, safety and immunogenicity
Radius Health Inc., of Waltham, Mass., and the Menarini Group, of Florence, Italy Elacestrant  Oral selective estrogen receptor degrader ER+/HER2-negative advanced or metastatic breast cancer The target enrollment milestone has been reached in the trial 


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